George Tachas, PhD, on Studying Proteomics of Antisense Oligonucleotides in DMD

"We're keen to understand what else the drug was doing. So, we used a proteomics approach... and we looked at 7000 different proteins in the blood. We collected patients’ blood at the end of the study and at various times during the study... So that was the designs, looking at the different protein changes during that period.”

ATL1102 modulated sVCAM-1, LTBP4, BMP5, BMP6, IGF-I, and Thrombospondin-1 closer to control levels (P <.0005), genetic modifiers of disease progression and fibrosis, in non-ambulant boys with Duchenne muscular dystrophy (DMD). ATL1102 is an antisense oligonucleotide and targets inflammation as a secondary cause of muscle damage in patients with DMD.

These data, from a proteomic analysis of plasma from participants in a phase 2 trial (NCT05938023) of ATL1102, were presented at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, by George Tachas, PhD, lead scientist, Percheron Therapeutics.

CGTLive® spoke with Tachas to learn more about the study and its design. He discussed how the study is analyzing the proteomics of blood samples collected from the participants at different timepoints throughout the study. He also shared that Percheron will be publishing a paper on the proteomics analysis and the full readout of the generated data, which include surprising findings on CTGF and fibrosis in patients with DMD.

Click here to view more coverage of the 2024 MDA Conference.

REFERENCE

Tachas G, DeLisle R, Mueller C, et al. ATL1102 treatment of non-ambulant boys with DMD stabilizes function modifying plasma proteins with roles in immune, fibrosis, bone & growth physiology. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL. Poster #V401